This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Lumacaftor and Ivacaftor (Orkambi) for Cystic Fibrosis Homozygous for the F508del Mutation in Patients Aged 6‐11 Years

Cystic fibrosis is an inherited disease that has severe effects on the lungs and the digestive system. It affects the cells that produce mucus and digestive juices. In cystic fibrosis, the secretions become thick and cause blockage within the tissues and organs (mostly within the lungs). Build‐up of thick and sticky secretions in the lungs causes inflammation and long‐term infection. In the gut, blockage of the tubes from the pancreas slows down the digestion of food and causes poor growth. People with cystic fibrosis are also at a higher risk of developing a number of related conditions such as diabetes, thin, weakened bones (osteoporosis) and liver problems, leading to a poor quality of life.
Lumacaftor and ivacaftor (Orkambi) is a medicine used to treat cystic fibrosis in patients aged 6 years and above who have a genetic mutation called the F508del mutation. This mutation affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR) which is involved in regulating the production of mucus and digestive juices. Orkambi is used in patients who have inherited the mutation from both parents and therefore have the mutation in both copies of the CFTR gene. Orkambi is taken orally as tablets twice a day. It offers a new treatment option for patients with cystic fibrosis with the advantage of targeting the actual disease process rather than just the symptoms.

Download Full Article

Twitter

Related Posts

Nintedanib is already licensed for treating idiopathic pulmonary fibrosis which is a subtype of ILDs. Nintedanib acts by blocking specific enzymes and pathways that lead to the development of blood vessels within lung cells involved in the scarring process. This inhibits further growth of the scarring tissues resulting in slowing of the disease progression. This action has been shown to be similar in patients with idiopathic pulmonary fibrosis. If licensed, nintedanib may provide the first novel therapy for PF-ILD where limited treatment options currently exist.

Cysteamine bitartrate as an oral formulation is a novel treatment of exacerbations of CF-associated lung disease, with a unique multi-action: breaking down of the excessive mucus produced by the lining of the airways, killing of the bacteria responsible for the recurrent respiratory infections, and disrupting the biofilms in which they colonise. Cysteamine bitartrate also potentiates the activity of standard of care antibiotics used in CF patients, and is potentially antibiotic-sparing as an adjunct treatment alongside standard of care therapy. If licensed, cysteamine bitartrate will offer an additional treatment option for adult patients with CF exacerbations.

Cystic fibrosis is an inherited disease that has severe effects on the lungs and the digestive system. It affects the cells that produce mucus and digestive juices. In cystic fibrosis, the secretions become thick and cause blockage within the tissues and organs (mostly within the lungs). Build-up of thick and sticky secretions in the lungs …

Nintedanib (OFEV®) is a medicine that is being developed for the treatment of Systemic Sclerosis associated interstitial lung disease (SSc-ILD). It acts by targeting the specific mechanisms by which scarring of the lungs occur, reducing progression of the disease. It is given by mouth as capsules. Nintedanib (as OFEV) is already available in the EU for the treatment of idiopathic pulmonary fibrosis (IPF). Because SSc-ILD and IPF share similarities in how the underlying lung scarring, or fibrosis, forms in people with the disease, the development of nintedanib for the treatment of SSc-ILD may address unmet needs. If licensed, nintedanib may offer an additional treatment option (the first licensed) for patients with SSc-ILD.